Craig Mayer, Nick WIlliams, Vojtech Huser
Lister Hill National Center for Biomedical Communications, National Library of Medicine, NIH, Bethesda, MD

See the full regCTG repository at https://github.com/lhncbc/CRI/tree/master/regCTG

To return to the list of conditions and view other reports go to https://lhncbc.github.io/CRI/regCTG/regCTG-condition_report_links.html

Study Counts

Type Study_count
Total_Studies 103
interventional 61
Observational 32
Registry 10

Studies over Time by Start Date

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Interventional Trials

Intervention Type

intervention_type Study_Count
Drug 23
Drug|placebo 12
Other 11
Biological 8
Device 3
Procedure 2
Behavioral 1
Drug|Procedure 1

Count of Studies by Intervention (normalized and mapped)

Interventions over Time

Yearly studies for each intervention by start date

Studies by Country

Country Study_Count
United States 21
France 4
Germany 4
NA 3
United States|Canada 3
Belgium|France|Germany|Italy|Netherlands|Poland|United Kingdom 2
Denmark 2
Italy 2
Netherlands 2
Belgium|Bulgaria|Denmark|Germany|Italy|Poland|Russian Federation 1
Belgium|France|Germany 1
Belgium|France|Italy|United Kingdom 1
Canada 1
Japan|Korea, Republic of|Taiwan 1
Taiwan 1
United States|Australia|Belgium|Brazil|China|Italy|Poland|Russian Federation|Saudi Arabia|Taiwan 1
United States|Australia|Belgium|Canada|France|Germany|Hong Kong|Italy|Japan|Korea, Republic of|Spain|Sweden|Turkey|United Kingdom 1
United States|Australia|Belgium|Canada|France|Italy|Japan|Korea, Republic of|Spain|Taiwan|United Kingdom 1
United States|Australia|Belgium|Canada|Germany|Israel|Italy|Japan|Korea, Republic of|Spain|Taiwan|United Kingdom 1
United States|Australia|Canada|Germany|Italy|Qatar|Taiwan|Turkey 1
United States|Belgium|Brazil|Canada|China|Croatia|France|Germany|Italy|Japan|Poland|Russian Federation|Serbia|Spain|Turkey 1
United States|Belgium|Brazil|China|Croatia|France|Italy|Japan|Poland|Russian Federation|Saudi Arabia|Serbia|Spain|Switzerland|Turkey 1
United States|Belgium|France|Germany|Italy|Netherlands|Poland|Switzerland|United Kingdom 1
United States|Canada|France|Germany|Hong Kong|Italy|Japan|Korea, Republic of|Spain|Sweden 1
United States|Canada|Germany 1
United States|Estonia|Latvia|Spain|Taiwan 1
United States|Italy|Netherlands|Spain 1

Sites per Study

Site_count Study_Count
1 32
2 1
3 1
4 6
6 3
8 1
9 1
10 1
14 1
15 1
16 2
19 2
22 1
24 3
29 1
30 1
35 1
42 1
43 1

Phase

Phase Study_Count
Phase 2 18
N/A 16
Phase 1 8
Phase 1/Phase 2 6
Phase 3 6
Phase 2/Phase 3 5
Early Phase 1 1
Phase 4 1

Number of Arms

Number_of_Arms Count_of_Studies
1 24
2 23
3 3
4 3
5 2
6 1
NA 5

Enrollment Metrics by Phase

Measure Early.Phase.1 N.A Phase.1 Phase.1.Phase.2 Phase.2 Phase.2.Phase.3 Phase.3 Phase.4
Min. 10 4.00 15.00 14.00 12.00000 11.00 6.00000 308
1st Qu. 10 9.75 24.00 27.50 20.25000 49.25 24.00000 308
Median 10 14.50 27.50 31.50 36.00000 93.50 31.50000 308
Mean 10 21.75 29.50 30.50 55.11111 107.25 84.83333 308
3rd Qu. 10 30.00 35.25 37.75 67.00000 151.50 102.75000 308
Max. 10 81.00 47.00 40.00 174.00000 231.00 292.00000 308

Trial Group Type

group_type Group_Count
Experimental 75
Placebo Comparator 13
Other 8
Active Comparator 7
NA 5
No Intervention 3
Sham Comparator 1

Intervention Model

intervention_model Study_Count
Single Group Assignment 32
Parallel Assignment 18
Crossover Assignment 6
Sequential Assignment 4
NA 1

Primary Purpose

primary_purpose Study_Count
Treatment 50
Diagnostic 3
Basic Science 2
Prevention 2
NA 2
Other 1
Supportive Care 1

Observational Studies

Studies by Country

Country Study_Count
United States 15
NA 3
Taiwan 3
United States|Canada 2
Belgium 1
Brazil 1
China 1
Korea, Republic of 1
Mali 1
Switzerland 1
Turkey 1
United Kingdom 1
United States|Germany|South Africa 1

Sites per Study

Site_count Study_Count
1 23
2 2
3 2
8 1
14 1
15 2
18 1

Enrollment Metrics

Measure Observational
Min 12.00
1st Qu 38.75
Median 135.00
Mean 30572.47
3rd Qu 700.25
Max 400000.00

Observation Model

observational_model Study_Count
Cohort 19
Case-Only 4
Other 3
Case Control 2
NA 2
Case-Control 1
Ecologic or Community 1

Time Perspective

time_perspective Study_Count
Prospective 26
Retrospective 3
Cross-Sectional 2
NA 1

Registries

Studies by Country

Country Study_Count
United States 6
France 2
Brazil 1
Egypt 1

Sites per Study

Site_count Study_Count
1 6
6 1
8 1
9 1
26 1

Enrollment Metrics

Measure Registries
Min 30.0
1st Qu 100.0
Median 200.0
Mean 620.9
3rd Qu 875.0
Max 3000.0

Registry Model

observational_model Study_Count
Case-Control 3
Cohort 3
Case-Only 2
Ecologic or Community 1
Family-Based 1

Time Perspective

time_perspective Study_Count
Prospective 5
Other 3
Cross-Sectional 2

Follow-up

target_duration Study_Count
1 Day 2
5 Years 2
1 Year 1
10 Years 1
15 Years 1
50 Years 1
6 Months 1
9 Years 1

Overview of Studies

Interventional Trials

#If less then 500 trials

nct_id brief_title link overall_status source primary_completion_date
NCT04042025 Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi https://ClinicalTrials.gov/show/NCT04042025 Enrolling by invitation AveXis, Inc. 2034-12-31
NCT03395795 Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II https://ClinicalTrials.gov/show/NCT03395795 Recruiting Assistance Publique - Hôpitaux de Paris 2020-02-29
NCT03819660 Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 https://ClinicalTrials.gov/show/NCT03819660 Enrolling by invitation Catalyst Pharmaceuticals, Inc. 2020-12-31
NCT03306277 Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 https://ClinicalTrials.gov/show/NCT03306277 Completed AveXis, Inc. 2019-11-12
NCT03223051 Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03223051 Completed Centre d’Investigation Clinique et Technologique 805 2019-11-22
NCT03032172 A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03032172 Active, not recruiting Hoffmann-La Roche 2021-12-31
NCT02941328 SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 https://ClinicalTrials.gov/show/NCT02941328 Completed UMC Utrecht 2018-01-31
NCT02913482 Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02913482 Active, not recruiting Hoffmann-La Roche 2019-11-14
NCT02876094 Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02876094 Recruiting Children’s Hospital of Eastern Ontario 2020-07-31
NCT02644668 A Study of CK-2127107 in Patients With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02644668 Completed Cytokinetics 2018-05-31
NCT02633709 A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers https://ClinicalTrials.gov/show/NCT02633709 Completed Hoffmann-La Roche 2016-08-04
NCT02628743 A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT02628743 Completed Hoffmann-La Roche 2018-12-18
NCT02594124 A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. https://ClinicalTrials.gov/show/NCT02594124 Active, not recruiting Biogen 2023-08-29
NCT04089566 Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT04089566 Recruiting Biogen 2022-09-26
NCT02391831 Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02391831 Completed Institut de Myologie, France 2018-06-30
NCT02386553 A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02386553 Active, not recruiting Biogen 2025-02-25
NCT02341053 Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02341053 Active, not recruiting Gillette Children’s Specialty Healthcare 2019-12-31
NCT02292537 A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT02292537 Completed Biogen 2017-02-20
NCT02268552 An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT02268552 Active, not recruiting Novartis 2020-07-23
NCT02122952 Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 https://ClinicalTrials.gov/show/NCT02122952 Completed AveXis, Inc. 2017-12-15
NCT02061189 Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT02061189 Active, not recruiting Assistance Publique - Hôpitaux de Paris 2020-04-30
NCT01302600 Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients. https://ClinicalTrials.gov/show/NCT01302600 Completed Hoffmann-La Roche 2013-10-31
NCT02052791 An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) https://ClinicalTrials.gov/show/NCT02052791 Completed Biogen 2017-01-31
NCT02003937 Aerobic Training in Patients With Spinal Muscular Atrophy Type III https://ClinicalTrials.gov/show/NCT02003937 Completed Rigshospitalet, Denmark 2010-09-30
NCT01981915 Optimum Insufflation Capacity in NMD https://ClinicalTrials.gov/show/NCT01981915 Completed Universität Duisburg-Essen 2013-02-28
NCT01862042 Palliative Care in Spinal Muscular Atrophy (SMA) 1 https://ClinicalTrials.gov/show/NCT01862042 Completed Assistance Publique - Hôpitaux de Paris 2018-06-11
NCT01839656 A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT01839656 Completed Biogen 2017-08-21
NCT01780246 An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) https://ClinicalTrials.gov/show/NCT01780246 Completed Biogen 2014-02-28
NCT01703988 An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT01703988 Completed Biogen 2015-01-31
NCT01645787 Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients https://ClinicalTrials.gov/show/NCT01645787 Completed Columbia University 2015-09-30
NCT01611610 Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) https://ClinicalTrials.gov/show/NCT01611610 Completed Institut de Myologie, France 2013-05-31
NCT01522079 Spinal Muscular Atrophy and Cardiac Autonomic Function https://ClinicalTrials.gov/show/NCT01522079 Completed Centro Universitário Augusto Motta 2011-08-31
NCT01494701 An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT01494701 Completed Biogen 2013-01-31
NCT04115475 New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT04115475 Active, not recruiting University of Erlangen-Nürnberg Medical School 2020-01-30
NCT04050852 Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments https://ClinicalTrials.gov/show/NCT04050852 Recruiting NYU Langone Health 2020-07-17
NCT03988907 A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants https://ClinicalTrials.gov/show/NCT03988907 Completed Hoffmann-La Roche 2019-09-29
NCT03970135 Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass https://ClinicalTrials.gov/show/NCT03970135 Completed Rigshospitalet, Denmark 2017-11-30
NCT03921528 An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03921528 Active, not recruiting Scholar Rock, Inc. 2021-01-31
NCT03920865 A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function https://ClinicalTrials.gov/show/NCT03920865 Completed Hoffmann-La Roche 2020-01-02
NCT03837184 Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies https://ClinicalTrials.gov/show/NCT03837184 Recruiting AveXis, Inc. 2021-04-01
NCT03781479 Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients https://ClinicalTrials.gov/show/NCT03781479 Recruiting Catalyst Pharmaceuticals, Inc. 2020-05-15
NCT03779334 A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03779334 Recruiting Hoffmann-La Roche 2021-06-21
NCT03505099 Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 https://ClinicalTrials.gov/show/NCT03505099 Active, not recruiting AveXis, Inc. 2021-06-24
NCT03461289 Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 https://ClinicalTrials.gov/show/NCT03461289 Active, not recruiting AveXis, Inc. 2020-09-30
NCT01233817 Progressive Strength Training in Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT01233817 Completed University of Utah 2013-06-30
NCT01166022 Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT01166022 Completed Columbia University 2014-08-31
NCT01028833 Effects of Power Mobility on Young Children With Severe Motor Impairments https://ClinicalTrials.gov/show/NCT01028833 Completed University of Oklahoma 2012-12-31
NCT02908685 A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants https://ClinicalTrials.gov/show/NCT02908685 Active, not recruiting Hoffmann-La Roche 2019-09-06
NCT02428673 Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions https://ClinicalTrials.gov/show/NCT02428673 Active, not recruiting Gillette Children’s Specialty Healthcare 2019-03-31
NCT04262570 Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) https://ClinicalTrials.gov/show/NCT04262570 Recruiting University of Erlangen-Nürnberg Medical School 2021-12-31
NCT00661453 CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I https://ClinicalTrials.gov/show/NCT00661453 Completed University of Utah 2012-05-31
NCT00568802 A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients https://ClinicalTrials.gov/show/NCT00568802 Completed Stanford University 2010-03-31
NCT00568698 A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients https://ClinicalTrials.gov/show/NCT00568698 Completed Stanford University 2012-02-29
NCT00533221 Pilot Study of Growth Hormon to Treat SMA Typ II and III https://ClinicalTrials.gov/show/NCT00533221 Completed University Hospital Freiburg 2010-02-28
NCT00528268 Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00528268 Completed University of Utah 2013-12-31
NCT00485511 A Trial of Hydroxyurea in Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00485511 Completed Kaohsiung Medical University Chung-Ho Memorial Hospital 2009-06-30
NCT00481013 Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00481013 Completed University of Utah 2009-12-31
NCT00374075 Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00374075 Completed University of Utah NA
NCT00324454 Levetiracetam for Cramps, Spasticity and Neuroprotection in Motor Neuron Disease https://ClinicalTrials.gov/show/NCT00324454 Completed Duke University 2007-12-31
NCT00227266 Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00227266 Completed University of Utah 2007-11-30
NCT00004771 Phase II Study of Leuprolide and Testosterone for Men With Kennedy’s Disease or Other Motor Neuron Disease https://ClinicalTrials.gov/show/NCT00004771 Completed Office of Rare Diseases (ORD) NA

Observational Studiess

#If less then 500 studies

nct_id brief_title link overall_status source primary_completion_date
NCT03300869 Natural History of Types 2 and 3 SMA in Taiwan https://ClinicalTrials.gov/show/NCT03300869 Recruiting Kaohsiung Medical University Chung-Ho Memorial Hospital 2019-12-31
NCT03217578 Neonatal Spinal Muscular Atrophy (SMA) Screening https://ClinicalTrials.gov/show/NCT03217578 Recruiting Kaohsiung Medical University Chung-Ho Memorial Hospital 2019-12-31
NCT02895789 Oxidative Capacity and Exercise Tolerance in Ambulatory SMA https://ClinicalTrials.gov/show/NCT02895789 Recruiting Columbia University 2020-04-30
NCT02852278 A Patient Centric Motor Neuron Disease Activities of Daily Living Scale https://ClinicalTrials.gov/show/NCT02852278 Completed University of South Florida 2019-09-13
NCT02532244 Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases https://ClinicalTrials.gov/show/NCT02532244 Recruiting Nemours Children’s Clinic 2022-12-31
NCT02466529 Natural History of Spinal Muscular Atrophy Type 1 in Taiwan https://ClinicalTrials.gov/show/NCT02466529 Completed Kaohsiung Medical University Chung-Ho Memorial Hospital 2015-12-31
NCT02327845 Phenotype, Genotype & Biomarkers in ALS and Related Disorders https://ClinicalTrials.gov/show/NCT02327845 Recruiting University of Miami 2021-06-30
NCT02123186 Newborn Screening for Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT02123186 Completed National Taiwan University Hospital 2016-10-31
NCT02044029 Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers. https://ClinicalTrials.gov/show/NCT02044029 Completed Hoffmann-La Roche 2015-07-31
NCT01931644 At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions https://ClinicalTrials.gov/show/NCT01931644 Recruiting Sanguine Biosciences 2025-08-31
NCT01910168 A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis https://ClinicalTrials.gov/show/NCT01910168 Completed Hoffmann-La Roche 2014-02-28
NCT01754441 Mechanisms of Cell Death in Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT01754441 Completed Nemours Children’s Clinic 2020-02-29
NCT01736553 Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development https://ClinicalTrials.gov/show/NCT01736553 Completed Ohio State University 2015-09-30
NCT04317794 Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) https://ClinicalTrials.gov/show/NCT04317794 Recruiting Biogen 2022-05-11
NCT04292574 UK Spinal Muscular Atrophy Patient Registry https://ClinicalTrials.gov/show/NCT04292574 Recruiting Newcastle University 2021-12-01
NCT04228718 The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs https://ClinicalTrials.gov/show/NCT04228718 Completed Marmara University 2020-02-28
NCT04193085 Wearable Technology to Assess Gait Function in SMA and DMD https://ClinicalTrials.gov/show/NCT04193085 Recruiting Columbia University 2020-12-31
NCT04010604 A Registered Cohort Study on SMA https://ClinicalTrials.gov/show/NCT04010604 Recruiting First Affiliated Hospital of Fujian Medical University 2039-12-31
NCT03912987 TRIAL READY (Clinical Trial Readiness) https://ClinicalTrials.gov/show/NCT03912987 Recruiting University of Miami 2022-01-31
NCT03878030 Effect of Nusinersen on Adults With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03878030 Active, not recruiting Northwell Health 2023-01-01
NCT03808233 Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03808233 Completed University of Texas Southwestern Medical Center 2018-06-01
NCT03709784 Spinraza in Adult Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03709784 Recruiting Washington University School of Medicine 2022-01-30
NCT03655223 Early Check: Expanded Screening in Newborns https://ClinicalTrials.gov/show/NCT03655223 Enrolling by invitation RTI International 2021-06-30
NCT03554343 Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium https://ClinicalTrials.gov/show/NCT03554343 Recruiting Centre Hospitalier Régional de la Citadelle 2021-03-04
NCT03489278 Clinical Procedures to Support Research in ALS https://ClinicalTrials.gov/show/NCT03489278 Recruiting University of Miami 2024-06-30
NCT03421977 Long-Term Follow-up Study for Patients From AVXS-101-CL-101 https://ClinicalTrials.gov/show/NCT03421977 Active, not recruiting AveXis, Inc. 2033-12-31
NCT01059240 SMN Copy Number Distribution in Mali, West Africa https://ClinicalTrials.gov/show/NCT01059240 Completed National Institutes of Health Clinical Center (CC) NA
NCT01033331 Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid https://ClinicalTrials.gov/show/NCT01033331 Completed University of Sao Paulo General Hospital 2008-08-31
NCT04139343 Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT04139343 Recruiting Ohio State University 2020-12-31
NCT00756821 A Pilot Study of Biomarkers for Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00756821 Completed HealthCore-NERI 2009-03-31
NCT00443066 Clinical Study of Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT00443066 Active, not recruiting Columbia University 2020-10-31
NCT00061607 Measuring Levels of SMN in Blood Samples of SMA Patients https://ClinicalTrials.gov/show/NCT00061607 Completed National Institutes of Health Clinical Center (CC) NA

Registries

#If less then 500 registries

nct_id brief_title link overall_status source primary_completion_date
NCT03339830 European Registry of Patients With Infantile-onset Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT03339830 Recruiting Institut de Myologie, France 2022-12-01
NCT02831296 Prospective Evaluation of Infants With Spinal Muscular Atrophy: https://ClinicalTrials.gov/show/NCT02831296 Recruiting Massachusetts General Hospital 2022-03-31
NCT02574390 Answer ALS: Individualized Initiative for ALS Discovery https://ClinicalTrials.gov/show/NCT02574390 Completed Johns Hopkins University 2019-12-31
NCT02124616 National Registry for Egyptian Pediatric Neuromuscular Diseases https://ClinicalTrials.gov/show/NCT02124616 Recruiting Ain Shams University 2020-04-30
NCT04177134 French Register of Patients With Spinal Muscular Atrophy https://ClinicalTrials.gov/show/NCT04177134 Recruiting Assistance Publique - Hôpitaux de Paris 2029-01-31
NCT04174157 Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) https://ClinicalTrials.gov/show/NCT04174157 Recruiting AveXis, Inc. 2038-06-30
NCT03706391 Study of ALS Reversals 4: LifeTime Exposures https://ClinicalTrials.gov/show/NCT03706391 Enrolling by invitation Duke University 2020-08-01
NCT03464903 Study of ALS Reversals 2: Genetic Analyses https://ClinicalTrials.gov/show/NCT03464903 Enrolling by invitation Duke University 2020-08-01
NCT04404764 Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study https://ClinicalTrials.gov/show/NCT04404764 Recruiting Hospital Israelita Albert Einstein 2020-12-31
NCT00466349 International SMA Patient Registry https://ClinicalTrials.gov/show/NCT00466349 Completed Indiana University 2018-01-31